As I sat down to write this bit of Insolence, I had at least two ideas for what I thought would be informative, entertaining, and timely posts. I also didn’t want to have to write about Stanislaw Burzynski again after having just done so on Friday, having to note that the FDA caved, granting compassionate use exemptions to use antineoplastons (ANPs) to several patients who had been lobbying for just such exemptions. I already described why what the FDA did was horrifically cynical. When I first saw the message rejoicing of the FDA’s decision in message on the Prayers for Elisha Facebook page, I was cautious. I could find confirmation of this news nowhere else, not even on the ANP Coalition webpage. Then, on Friday afternoon, Liz Szabo, the USA TODAY reporter who previously published her epic expose of the Burzynski cancer machine back in November, basically confirmed the report with a followup story in USA TODAY, FDA agrees to let patients get controversial drug:

The Food and Drug Administration has agreed to allow a handful of cancer patients to receive unapproved drugs from a controversial Texas doctor, but only if they can find another physician to administer them.

The drugs are made by Houston doctor Stanislaw Burzynski, who was the subject of a USA TODAY investigation last year. While his supporters consider him a medical maverick, mainstream doctors describe him as a snake-oil salesman. Burzynski has claimed for more than 36 years to be able to cure certain hard-to-treat brain tumors with drugs he calls antineoplastons. The National Cancer Institute notes that Burzynski has never published definitive evidence that his drugs cure cancer or even help people live longer.

Burzynski has been unable to give these drugs since 2013, when the FDA placed his experiments on hold after the death of a 6-year-old New Jersey boy taking antineoplastons. In December, the FDA sent Burzynski a warning letter, noting that he inflated his success rates and failed to report side effects and to prevent patients from repeatedly overdosing. The Texas Medical Board also charged Burzynski last year with false advertising.

She also confirmed that the FDA had caved:

The FDA acknowledged Friday that it has agreed to allow them to use the experimental drug, but only if they can find a qualified, independent physician to administer the drug. Beyond infusing the drug and overseeing their care, the doctor would have to formally apply for expanded access to an “investigational new drug,” as well as get approval from an institutional review board, an independent panel that reviews safety and ethical issues involved in clinical trials.

Over the weekend, supporters of Stanislaw Burzynski wasted no time in using the FDA decision to defend Burzynski and, ironically, to attack the FDA. Many of you might remember McKenzie Lowe, the 12-year-old girl diagnosed with diffuse intrinsic pontine glioma (DIPG) in 2012. Her family has been trying to have her treated by Stanislaw Burzynski, making videos and lobbying Congress. Indeed, they succeeded in persuading their senator, Senator Kelly Ayotte (R-NH), to write to the FDA urging it to consider allowing McKenzie to be treated by Burzynski. On a Facebook page Friends of McKenzie Lowe, a message was posted on Sunday simultaneously rejoicing at the news as evidence that McKenzie has a chance to live and lambasting Liz Szabo:

As you all probably have read in the USA Today the FDA has agreed to let Mckenzie and the other people who want to use ANP CAN USE IT!!!! FANTASTIC!!!!! But as you can see the writer of the article is very bias on the negative side. As usual she one again took something good for these patients and in stead of focusing on the positive response from the FDA she took another swing at bashing Dr. Burzynski and his clinic. There was no mention of or comments from Anthony Stout, Rick Schiff, Jessica Ressel, Randy Hinton, Philip Norton and many many more..

Let’s go back in history to all the nay Sayers that said Christopher Columbus was crazy. The world is flat, you will fall off the end. Hmmm. Einstein was a quack… Hmmm. Isaac Newton and Galileo were also shunned by their peers… Thank god they had the fortitude to push aside the nay sayers and continue to prove them ALL wrong. Imagine where we would ALL be today if they had listened to their critics and not persevered with what they believed.

Unfortunately, this is known as the Galileo Gambit, a nice, concise term used to argue that if you are vilified for your ideas it must mean that you are right. Unfortunately, what supporters of pseudoscientists and quacks frequently forget is that there are two requirements necessary to be able to credibly claim the mantle of Galileo. Not only must you be vilified, but you must be correct. Only one of these applies to Stanislaw Burzynski, and it isn’t the second one.

As has been the case whenever I’ve discussed patients like McKenzie Lowe, Liza Covad-Hauser, Rafael Elisha Cohen, and others, I understand, at least as much as it is possible to understand without actually being in their shoes. As I’ve pointed out before, it was only five years ago that my mother-in-law died of widely metastatic breast cancer. Burzynski couldn’t have saved her. ANPs couldn’t have saved her. And I know I’m going to take some flak for saying this, but ANPs cannot save McKenzie Lowe. At least, there is no convincing evidence that they can, and, quite frankly, the only blame for why this is true should be placed at the feet of Stanislaw Burzynski himself. He’s the one who, thinking himself the “brave maverick doctor” to whom the rules of science don’t apply, has failed to produce such evidence. While I understand the desperation these families are feeling, I keep hoping that they will realize that it is not doing McKenzie, Elisha, or any of the others any favors to subject them to toxic chemotherapy—and, make no mistake, ANPs are toxic chemotherapy, efforts of advocates to paint them as “natural” and “nontoxic” notwithstanding. As I’ve pointed out time and time again: If there’s anything worse than dying of a terminal illness, it’s dying of a terminal illness and suffering in the process unnecessary complications or pain for no benefit.

I can totally understand why families like the Lowes might become very excited about the possibility of being able to use ANPs. As misled as they have been and mistaken as their belief is, they do, after all, genuinely believe that ANPs represent the last chance their loved ones have to be able to survive their cancer. Unfortunately, our old “friend” and Burzynski’s very own propagandist, Eric Merola, who has produced two fawning “documentaries” about the Great Man chock full of misinformation and exaggerations, coupled with attacks on Burzynski critics and skeptics, has no such excuse and deserves none of the compassion that we should all have for patients dying of brain tumors and the families who love them. Not surprisingly, he, too, has leapt into the fray with a typically frothing-at-the-mouth conspiracy-laden pile of fetid dingos’ kidneys of a response to Liz Szabo’s story, in which he examines it line-by-line and basically responds with typical Burzynski propaganda, misinformation, and lies.

There’s too much there for a full response in this post. Besides, most of the misinformation that’s there I’ve covered before, often multiple times. Perhaps I’ll respond a bit more on my not-so-super-secret other blog. Here, I simply want to point out a few of the most egregious examples. For example, here is probably the most egregious lie on Merola’s part:

A DIPG in a child has never been cured in medical history — why would the FDA make such a claim in this case? It’s quite simple, the science proved it works and cures in many cases, and therefore the science was allowed to progress. There are no “claims” other than what can be proven. Maybe it’s because the cancer industry doesn’t like anyone else “claiming” anything as they own the luxury of claiming what cancer medications work or not work — and we know how well that has worked out for us.

As I’ve pointed out before, this is, quite simply, untrue. It’s also been pointed out time and time again to Merola that this is untrue. Long term survival for patients with DIPG is rare, definitely, but it’s always dangerous to make an absolute statement like the one that Merola has done. Contrary to Burzynski’s claim, there are occasional long-term survivors, some of whom received little or no therapy, such Connor Frankenberg, a child from Germany, and two patients who underwent spontaneous remission. There is even a site, the DIPG Registry, whose purpose is to report on how patients with DIPG do. and promoting Just One More Day, a registered non-profit 501(c)(3) organization dedicated to helping families affected by a diffuse intrinsic pontine glioma, established by families of DIPG victims. The DIPG Registry concludes that, although the vast majority of children with DIPGs do poorly:

The bottom line is: 1) we really don’t understand DIPGs, and 2) there are children who have been diagnosed with DIPGs-based on the incomplete knowledge that we currently have- who truly have had spontaneous regression of their tumors and 3) most of the children with reports of spontaneous regression have been very young.

The big problem is that Burzynski appears not to keep good enough records to demonstrate one way or the other whether he can cure DIPG, and we already know from recent FDA findings and Liz Szabo’s USA TODAY report that he has misclassified partial and complete responses. Yes, DIPG is a horrible disease, with only 10% surviving beyond two years. Unfortunately, there is no convincing evidence that Burzynski can do any better than conventional oncology, much less cure DIPG, as much as his propagandist Eric Merola repeats the claim that he can. Worse, Burzynski hasn’t published a single one of his completed phase II clinical trials, even though he’s had well over 15 years to complete them. With tumors this lethal, most such trials should be completed within five years, which brings us to more of Merola’s misinformation:

The National Cancer Institute notes that Burzynski has never published definitive evidence that his drugs cure cancer or even help people live longer.

The above links are examples definitive published evidence. Notice how this writer links to a PDF highlighting her point, vs. the National Cancer Institute (NCI) website itself. Is it perhaps because on the NCI website it clearly says:

“A phase II study also conducted by the developer [Burzynski] and his associates at his clinic reported on 12 patients with recurrent and diffuse intrinsic brain stem glioma. Of the ten patients who were evaluable, two achieved complete tumor response, three had partial tumor response, three had stable disease, and two had progressive disease. Patients ranged in age from 4 to 29 years.”

Merola clearly does not understand the difference between “responses” and cures. Similarly, he does not understand what constitutes “definitive evidence.” (Hint: It isn’t case reports, tiny case series published in bottom-feeding journals, or incomplete phase II trials. Moreover, just because the NCI cited a crappy review article by Burzynski that reported partial results phase II trials, a ten year old partial report of a phase II trial, a dubious paper published in an integrative medicine journal, and another review article published by Burzynski in a journal that appears not to be even indexed by PubMed does not constitute “definitive evidence” that ANPs work. All it means is that the NCI was examining the existing published evidence on its way to writing this:

To date, no randomized controlled trials examining the use of antineoplastons in patients with cancer have been reported in the literature. Existing published data have taken the form of case reports or series, phase I clinical trials, and phase II clinical trials, conducted mainly by the developer of the therapy and his associates. While these publications have reported successful remissions with the use of antineoplastons, other investigators have been unable to duplicate these results [10] and suggest that interpreting effects of antineoplaston treatment in patients with recurrent gliomas may be confounded by pre-antineoplaston treatment and imaging artifacts.[11,14,16] Reports originating from Japan on the effect of antineoplaston treatment on brain and other types of tumors have been mixed, and in some Japanese studies the specific antineoplastons used are not named.[9] In many of the reported studies, several or all patients received concurrent or recent radiation therapy, chemotherapy, or both, confounding interpretability.

Mr. Merola, that’s hardly “definitive”! Nor is the “definitive” study by Hidaeki Tsuda featured in the second Burzynski movie, given that nothing has been published to allow us to evaluate the methods. Nor is the fact that The Lancet Oncologyapparently editorially rejected a manuscript submitted by Burzynski evidence of any sort of “conspiracy” against him. Indeed, I now probably know why Burzynski’s manuscript was rejected. Look at the title: Glioblastoma multiforme: A report of long-term progression-free and overall survival of 8 to over 16 years after antineoplaston therapy and a review of the literature. Yes, the title seems to indicate that this is just another small case series. No wonder The Lancet Oncology didn’t even bother to send it out for review!

Of course, Merola can’t resist a swipe at yours truly, who was quoted in the article:

A guy who is paid to write blogs attacking this innovation and other innovations that compete with the status quo, a guy who has never once met a Burzynski patient, never once visited the Burzynski clinic, a guy who supposedly finished his residency and now writes “thought police” blogs for a living — yeah, let’s hear more about what this guy has to say, that’s productive. He does fit the angle for Liz’s one-sided propaganda, so I understand why she included his irrelevant opinion.

I don’t know whether to laugh or be annoyed at Merola’s continued lies about me. Once again, I do not need to visit the Burzynski Clinic to know that ANPs almost certainly don’t work. I did finish my residency and am board certified in surgery. If Mr. Merola doesn’t believe that, he can go to the American Board of Surgery website and search for my name in the section that lets the public verify the board certification of surgeons, realizing that you can’t become board certified without finishing an accredited residency. Finally, I don’t actually blog for a living. It’s my hobby. I’m not paid at all for my work at my other blog, and here I make a small amount of money that I could completely do without if this gig ever went south and I decided to bolt. As for having something worthwhile to say, well, let’s just say that I’d put my record for science-based analysis against Mr. Merola’s record for pseudoscience-based propaganda any day, particularly in light of his latest paean to another brave maverick, this time promoting the long-discredited cancer quackery known as laetrile.

What is puzzling to me is why the FDA caved. While it is true that the ANP Coalition managed to get a few misguided legislators, the most prominent of whom was Senator Kelly Ayotte (R-NH), to write letters to the FDA urging that it consider allowing ANPs to be made available through single patient IND/expanded access to their constituents, the letters were fairly wishy-washy. Moreover, the Change.gov petition to the White House response to a We The People online petition to have the FDA allow Elisha Cohen access to ANPs through compassionate use exemption resulted in a response that, boiled down to it essence, said that it’s the FDA’s job to make such decisions, and let the FDA do its job, along with links to various FDA web pages with information about investigational drugs and information about how the FDA could be contacted. It was a shockingly reasonable response.

One rationale reported in Szabo’s article seems to be that by allowing ANPs to be used but excluding Burzynski from having anything to do with administering them other than producing them, in essence, relegating him to the role of a drug manufacturer, useful data might be obtained and patients protected while obtaining that useful data. This is utter nonsense. As Howard Ozer is quoted in Szabo’s article:

“This is a cop-out,” says Ozer, director of the University of Illinois Cancer Center, who studied Burzynski’s research results in the 1990s.

“Because it can be toxic and cause life-threatening sodium problems, patients (who take antineoplastons) are at risk and could die,” Ozer says.

The scientific community will learn nothing by giving these patients antineoplastons, because there will be no comparison group, Ozer says. Any doctor who agrees to give antineoplastons could be biased in favor of them, which could skew the results.

Exactly. As Dr. Peter Adamson points out in the article, after 37 years, if antineoplastons were the wonder drugs that Burzynski claims them to be, scientists would know it by now. ANPs aren’t wonder drugs. There isn’t even any evidence that they’re better than standard-of-care, as dismal as that is for DIPG.

It is possible that one of the Senators or other legislators did indeed pressure the FDA, leading the relevant enforcement officials to come up with this decision in order to get them off their backs without actually doing anything. They could credibly say, “Hey, we offered compassionate use, but I’m sure you’ll agree that, given his history, we can’t let Burzynski be the one to administer the ANPs on compassionate use. Look at his history of playing fast and loose with IRBs and not obeying the rules with respect to INDs.” This might even sound convincing and seem clever, which is why I rather suspect that Dr. Henry Friedman is right when he says:

“It will deflect criticism from anybody who says the FDA is unilaterally denying dying patients something that could help them,” says Friedman, deputy director of the Preston Robert Tisch Brain Tumor Center at Duke University in North Carolina, who also reviewed Burzynski’s studies in the 1990s. “It will get FDA off the hook.”

That, I believe, is exactly why the FDA did it, not to help dying children. Unfortunately, the consequences of the FDA’s decision are likely to be disastrous. Long-suffering families will have false hope extended to them once again, only to find out that it’s a useless offer. In addition to continuing to scramble to raise money, they’ll scramble to find oncologists willing to do the not inconsiderable work to get a single patient IND approved and administer ANPs, something few, if any, of them are likely to succeed at doing. The families don’t know it yet, but what the FDA has done will only add to their misery. Indeed, I’ve already seen reports that patients have contacted “dozens” of pediatric oncologists looking for one who will administer ANPs, and, given that the number of pediatric oncologists in the US is small, a significant proportion of them are likely to be contacted.

Meanwhile, Burzynski gets a propaganda victory, thanks to the FDA’s failure to enforce its own standards, and alt-med proponents get another conspiracy theory to add to the list of conspiracy theories surrounding Burzynski already.

Related

Comments

I asked myself, how can the people still belive ANPs if they learn that for last 38 years Burzynski failed to get results published and the drug itself FDA approved and in wide-spread use. And then I remembered the conspirational narrative that is such great excuse. Which saddens me greatly, since as (hypothetical, sadly) head of Evil Big Pharma Cancer Industry if I learned about Brave Maverick Doctor and his Miracle Drug, I’d be doing my best to lay my hands on it and make filthy lucre out of curing cancer. Really people should more often imagine themselves in the role of conspirator.

But I guess that’s why I am cynical and disillusioned. (And in spirit of this: http://xkcd.com/1274/ hoping for better brand of conspiracy ruling the world.)

Also, if any of the people who will now set out to get ANPs off the FDA’s cave in, manages to get them and still dies, we all know the blame will be shifted on FDA for making them jump to the hoops till it was too late. Because miracle cure not being miraculous (or even curative at all) is less likely explanation that malevolence of administration. Conspirational Occam’s Razor anyone?

I wonder what the overlap area would be in the Venn diagram with one circle being the fanatic defenders of Burzynski’s “natural”, “non-toxic” potions (that are killing people from hypernatremia), and the other circle being the anti-sodium Nazis (who think ordinary table salt is somewhat more harmful than plutonium)?

HDB@3 — Newton tends not to get as much press these days as Einstein, but I think most physicists think of Newton as smarter. He may have been the smartest person who ever lived.

And, as you point out, his peers held him in awe. He once submitted an anonymous solution to a mathematical puzzle (the brachistochrone problem) that Bernoulli had posed; Bernoulli recognized it as Newton’s work with the wonderful phrase, “We know the lion by its claw”.

While we’re discussing the “mavericks” who were scoffed at, it’s worth pointing out that the criticism of Columbus had nothing to do with flat vs. round Earth (scholars knew the Earth was round), but rather about his calculations of the size of the Earth. And you know what? His detractors were correct. Columbus totally screwed up and was, in fact, wrong. It was simply a fortunate accident that he stumbled onto the Americas.

Let’s go back in history to all the nay Sayers that said Christopher Columbus was crazy. The world is flat, you will fall off the end. Hmmm.

The Galileo Gambit with a side of the Christopher Columbus myth and bee impersonations. The “nay sayers” were correct since not only did they know the world was round, they knew it was 3 times as big as Columbus though it was. Why people celebrate idiots who got lucky is beyond me.

Liz Szabo’s article reports that Liza Covad-Lauser has apparently already found an oncologist to do this and speculated that, for purposes of finding someone, being the wife of a rock star’s drummer helps, particularly when that rock star has put his name behind the campaign to let her use ANPs. As for the rest, I would hope that the pediatric oncologists out there would be more savvy. I have seen a couple of reports of patients lamenting that they’ve contacted “dozens” of pediatric oncologists without success. My guess is that none of them will be likely to find anyone, although it’s possible that one or two might. You have to remember that doing all the paperwork to do a single patient IND is a huge undertaking for an oncologist. No one who doesn’t strongly believe in ANPs is likely to be willing to do it. There’s also the expense. Burzynski might be supplying the ANPs for free, but all the other care is not going to be paid for by insurance and, if Burzynski’s not doing it, given that Burzynski promised to care for these eight or nine patients for free the families will have to pay out of pocket for everything except the ANPs.

I have seen a couple of reports of patients lamenting that they’ve contacted “dozens” of pediatric oncologists without success

I would be interested to hear what the conversations were. Did the oncologists say why they refused (an expensive, exhausting, and potentially harmful effort with practically no chance of success)? Did the parents listen?

Newton may not be the best example; as an individual he was what you across the water would probably call an a******* and he spent quite a lot of time working on what was essentially alchemy. Maybe that does make him a good example . . .

[…] posts about the utter failure of the FDA to do its job protecting cancer patients from the likes of Stanislaw burzynski, it’s time to move on. Given how utterly demoralizing it was to see the FDA, in essence, pass […]

Semmelweis: you want me to deliberately treat some patients with filthy hands in exchange for gaining legitimacy in the eyes of my medical peers?

Orac: Collateral damage is part of the beauty of the Scientific method.

Semmelweis : But that would be unethical!

Orac: you want me to stop bashing you on my blog dont you?

Semmelweis: i refuse to deliberately infect my patients with germs by not washing my hands.

Orac: Look, your ‘germ theory’ is imaginative fantasy. There is no such thing as tiny organisms we cant see infecting people. …. Now maybe if you conduct a random double blind study where only *a few* are infected we could get somewhere….

Actually, if Burzynski could really do as he claims, it would be fairly easy to prove, without randomized clinical trials.

The natural course of a disease like DIPG is well-documented. If Burzynski produced the evidence that he treated, say, a total of 100 DIPG patients and 25 of them survived for ten years or more, that would be wildly unlikely unless he really had a cure. Of course, in order for this to prove anything, he’d have to show that the 100 patients were not cherry-picked — that he didn’t have 5,000 such patients of which he picked the 100 best cases, 25 of whom (0.5% of the total) survived for ten years. Also, he would have to have not destroyed the records, so that one could be sure that they really had DIPG in the first place.

Okay, so it wouldn’t be so easy for Burzynski. But if an ethical and competent researcher had been running those clinical trials, it would have been.

[…] posts about the utter failure of the FDA to do its job protecting cancer patients from the likes of Stanislaw burzynski, it’s time to move on. Unfortunately, the first thing that caught my eye as I sat down to […]

It really frustrates me when people look at something like this and say “what’s the harm?” for conditions that sadly, we simply don’t have much to offer for yet. Especially with children. Even aside from the potential acute risks, it just seems so tragic to spend time and emotional energy on this crap instead of making the most of whatever time is left.

I started developing chronic pain and autoimmune symptoms when I was 18, and my mom spent a long time chasing after some kind of perfect cure. And honestly, it made it harder for me to deal, because I felt like I was disappointing her each time something new and “harmless” failed to work. And that was for a non-fatal, non-life-threatening, relatively manageble set of symptoms. I really worry about the pyschological effect of this stuff on child patients.

[…] Weekends seem to be the time for shameless self-promotion. At least, some weekends are. So, in that tradition, I can’t help but blowing my own horn a bit and urging my readers to head on over to listen to The Skeptics’ Guide to the Universe, Episode #455, March 29, 2014. There, Bob Blaskiewicz and I are interviewed about Stanislaw Burzynski and what you, as supporters of science-based medicine, can do to counter the efforts of his supporters to pressure the FDA to let children with cancer receive antineoplastons, efforts that are yielding fruit. […]

[…] despicable the Burzynski propaganda machine is, though, I can’t help but point out that it is still going full tilt. Just yesterday, as the news of Laura Hymas’s death was hitting Twitter and Facebook, the […]

[…] how despicable the Burzynski propaganda machine is, though, I can’t help but point out that it is still going full tilt. Just yesterday, as the news of Laura Hymas’s death was hitting Twitter and Facebook, the […]